Tafamidis: Difference between revisions
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<StructureSection load='' size='340' side='right' caption=' | <StructureSection load='' size='340' side='right' caption='Tafamidis' scene='10/1006890/Cv/2'> | ||
Tafamidis, sold under the brand names Vyndaqel and Vyndamax,<ref name="a4">[https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=1b4121ee-a733-4456-a917-be2603477839 "Vyndaqel- tafamidis meglumine capsule, liquid filled Vyndamax- tafamidis capsule, liquid filled". DailyMed. 30 August 2019. Retrieved 24 November 2019]</ref> is a medication used to delay disease progression in adults with certain forms of transthyretin amyloidosis. It can be used to treat both hereditary forms, familial amyloid cardiomyopathy and familial amyloid polyneuropathy, as well as wild-type transthyretin amyloidosis, which formerly was called senile systemic amyloidosis. It works by stabilizing the quaternary structure of the protein transthyretin. In people with transthyretin amyloidosis, transthyretin falls apart and forms clumps called (amyloid) that harm tissues including nerves and the heart.<ref name="a5">[https://www.ema.europa.eu/en/medicines/human/EPAR/vyndaqel"Vyndaqel EPAR". European Medicines Agency. 16 October 2019. Retrieved 24 November 2019.]</ref><ref name="a6">[https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatments-heart-disease-caused-serious-rare-disease-transthyretin-mediated "FDA approves new treatments for heart disease caused by a serious rare disease, transthyretin mediated amyloidosis". U.S. Food and Drug Administration. 14 September 2019. Archived from the original on 14 September 2019.]</ref> | Tafamidis, sold under the brand names Vyndaqel and Vyndamax,<ref name="a4">[https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=1b4121ee-a733-4456-a917-be2603477839 "Vyndaqel- tafamidis meglumine capsule, liquid filled Vyndamax- tafamidis capsule, liquid filled". DailyMed. 30 August 2019. Retrieved 24 November 2019]</ref> is a medication used to delay disease progression in adults with certain forms of [[transthyretin]] amyloidosis. It can be used to treat both hereditary forms, familial amyloid cardiomyopathy and familial amyloid polyneuropathy, as well as wild-type transthyretin amyloidosis, which formerly was called senile systemic amyloidosis. It works by stabilizing the quaternary structure of the protein transthyretin. In people with transthyretin amyloidosis, transthyretin falls apart and forms clumps called (amyloid) that harm tissues including nerves and the heart.<ref name="a5">[https://www.ema.europa.eu/en/medicines/human/EPAR/vyndaqel"Vyndaqel EPAR". European Medicines Agency. 16 October 2019. Retrieved 24 November 2019.]</ref><ref name="a6">[https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatments-heart-disease-caused-serious-rare-disease-transthyretin-mediated "FDA approves new treatments for heart disease caused by a serious rare disease, transthyretin mediated amyloidosis". U.S. Food and Drug Administration. 14 September 2019. Archived from the original on 14 September 2019.]</ref> See also [https://en.wikipedia.org/wiki/Tafamidis Tafamidis]. | ||
Tafamidis is a pharmacological chaperone that stabilizes the correctly folded tetrameric form of the transthyretin protein by binding in one of the two thyroxine-binding sites of the tetramer. In people with familial amyloid polyneuropathy, the individual monomers fall away from the tetramer, misfold, and aggregate; the aggregates harm nerves.<ref name="a8">PMID:22378262</ref> | |||
The maximum plasma concentration is achieved around two hours after dosing; in plasma it is almost completely bound to proteins. Based on preclinical data, it appears to be metabolized by glucuronidation and excreted via bile; in humans, around 59% of a dose is recovered in feces, and approximately 22% in urine.<ref name="a3">[https://www.medicines.org.uk/emc/product/2837 "Vyndaqel 20 mg soft capsules - Summary of Product Characteristics". Electronic Medicines Compendium. Retrieved 2 April 2018]</ref> | |||
<scene name='10/1006890/Overall/1'>Wild-type transthyretin in complex with tafamidis</scene> ([[3tct]]). | <scene name='10/1006890/Overall/1'>Wild-type transthyretin in complex with tafamidis</scene> ([[3tct]]). | ||